- a pilot study for patients with single system MFB and localized "SPECIAL SITES"

Description

Therapy for "LOW RISK" Patients:

The decision as to which research program you will be assigned will be made entirely by
chance. The overall time of therapy will be 6 or 12 months as randomly assigned. The
research program will be with the drugs Vinblastine and Prednisone.

Initial Therapy

1. Prednisone given by mouth three times a day daily as a four-week course, then gradually
decreased over 2 more weeks.

2. Vinblastine will be given IV (into a vein) one day a week for 6 weeks.

3. Patients who have no evidence of active disease at this time will proceed to
continuation therapy.

3. Prednisone in 3 divided doses days 1-3 weekly from week 7-12. 4. Vinblastine IV one day a
week for 6 more weeks.

- If the disease is gone or better after this additional therapy continuation will begin.

Continuation Therapy 3. Prednisone in 3 doses daily day 1-5 every 3 weeks until the end of
month 6. 4. Vinblastine IV day 1 every 3 weeks until the end of month 6.

Group 1 "RISK" patients:

The primary aim of the study is to compare the therapeutic efficacy of control arm A
(PDN+VBL) with the experimental arm B (PDN+VBL+MTX). The primary endpoint is the proportion
of non-responder in risk organs to the initial treatment.

Non-response to initial therapy is defined as:

• death within 12 weeks of initial treatment or

- progression (worse) in risk organs at week 6

- lack of response (=intermediate response or progression) in risk organs at week 12 as
compared to the status of disease at week 6.

If the null hypothesis is true, the two randomized treatment arms are equally effective in
terms of non-response. If the alternative hypotheses is true, there is a difference between
the two randomized arms in terms of efficacy.

Group 2 "LOW RISK" patients:

The primary aim of the study is to compare the reactivation free survival rate in initial
responders at week 6 with continuation treatment for 6 months (Arm LR 6) versus 12 months
(Arm LR 12) in those patients without disease reactivation within the first 6 months.

If the null hypothesis is true, the reactivation rate of both randomized arms are equal. If
the alternative hypothesis is true, there is a difference between the two arms in terms of
reactivation frequency.

Therapy for "RISK" Patients:

Treatment A will consist of:

7. Initial Therapy 8. Prednisone given by mouth three times a day daily as a four-week
course, then gradually decreased over 2 more weeks.

9. Vinblastine will be given IV (into a vein) one day a week for 6 weeks. 10. Patients who
have no evidence of active disease at this time will proceed to continuation therapy.

5. Prednisone in 3 divided doses by mouth for 3 days every week, from week 7-12.

6. Vinblastine IV one day a week for 6 more weeks.

** If the disease is gone or better after this additional therapy continuation will begin.

Continuation Therapy:

5. 6-MP by mouth daily until the end of month 12. 6. Prednisone in 3 doses daily day 1-5
every 3 weeks until the end of month 12. 7. Vinblastine IV day 1 every 3 weeks until the end
of month 12.

** Those patients whose disease didn't respond to the initial therapy by the 12th week will
come off this study and proceed to other research programs.

Treatment B will consist of:

1. Initial Therapy

2. Prednisone given by mouth three times a day daily as a four-week course then gradually
decreased over 2 more weeks.

3. Vinblastine will be given IV one day a week for 6 weeks.

4. Methotrexate given as a 24 hour IV infusion day 1 of weeks 1, 3, and 5, followed by
leucovorin.

5. Leucovorin is a drug that will be given to help the body remove the methotrexate and
decrease the possible side effects. (This is sometimes called a "leukovorin rescue".
The drug will be given by mouth.)

- Patients who have no evidence of active disease at this time will proceed to
continuation therapy.

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Medical and Biotech [MESH] Definitions

Prednisone

A synthetic anti-inflammatory glucocorticoid derived from CORTISONE. It is biologically inert and converted to PREDNISOLONE in the liver.

Procarbazine

An antineoplastic agent used primarily in combination with mechlorethamine, vincristine, and prednisone (the MOPP protocol) in the treatment of Hodgkin's disease.

Leucovorin

The active metabolite of FOLIC ACID. Leucovorin is used principally as its calcium salt as an antidote to folic acid antagonists which block the conversion of folic acid to folinic acid.

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